​Cancer Policy Monitor: August 14, 2018          

Register Today: Rally for Medical Research 2018   

Registration is now available for the sixth annual Rally for Medical Research, which will be held Sept. 12-13, 2018, in Washington, D.C. For those who cannot participate in person, the online Rally National Day of Action will take place Sept. 13. Stay tuned to the rally website for more info on the National Day of Action.

We have reserved a block of rooms at the rally headquarters hotel at a negotiated rate. Contact to make a reservation at the lower rate.

Stay up-to-date on registration information by visiting the rally website, Facebook, and Twitter pages. Learn more about 2018 Hill Day Sponsorship Opportunities.

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Appropriations Update from Capitol Hill   

Full Senate plans to consider the Labor-HHS-Education bill on the floor mid-August

The House of Representatives has adjourned for its five-week August recess, and members will not return to Washington until after Labor Day. However, the Senate will recess for just one week (Aug. 6-10) and return to Washington to continue its work on several legislative items, including the fiscal year (FY) 2019 appropriations bills.

The Senate is poised to bring the Labor, Health and Human Services (HHS), Education bill to the floor the week of Aug. 13. Senate leadership plans to pair Labor-HHS-Education with the largest spending bill, the Department of Defense, as they see this as the best possibility to gain broad bipartisan support for passage of both measures. 

The Senate version of the Labor-HHS-Education bill, which received strong bipartisan support, proposes a $2 billion increase for the NIH in FY 2019, for a total proposed funding level of $39.1 billion. The bill also includes a funding level for the National Cancer Institute (NCI) that is $6.147 billion, which includes $400 million designated for the Beau Biden Cancer Moonshot in FY 2019. This is a $190 million increase above FY 2018 levels.

The House Appropriations Committee passed its Labor-HHS-Education spending bill along party lines July 11. The bill includes a $1.25 billion increase for the NIH in FY 2019. House Subcommittee Chairman Tom Cole (R-OK) has indicated that as in recent years, Congress would likely agree on an NIH funding level closer to the Senate proposal. Chairman Cole was provided with a smaller overall budget allocation than what his Senate colleagues were given to disseminate across the many programs they oversee, which accounts for the lower proposed increase for NIH in the House bill. 

If the Senate passes the Labor-HHS bill later this month, the next steps remain unclear. While the House has passed several, less controversial appropriations bills, the Labor-HHS-Education bill would face significant political hurdles to get to the floor. If the bill passes on the House floor, The House and Senate also will have to convene a conference committee to resolve differences in the two versions of the bill.

Senate passes the FDA/Agriculture bill with full funding for the FDA Oncology Center of Excellence

On Aug. 1, the Senate voted 92-6 to approve a spending package that included the FY 2019 Agriculture, Rural Development, Food and Drug Administration and Related Agencies bill. The bill includes $2.97 billion in base funding for the FDA, a $159 million (5 percent) increase over the FY2018 enacted level. Total funding, when taking into account user fee revenues, would be $5.4 billion. The bill fully funds the Oncology Center of Excellence (OCE) at $20 million. The House has yet to pass the Agriculture/FDA spending bill, but its bill also includes a significant boost for the FDA and full funding for the OCE.

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FDA Announces Biosimilar Action Plan    

On July 18, U.S. Food and Drug Administration (FDA) Commissioner Scott Gottlieb, MD, announced the release of the agency's Biosimilar Action Plan (BAP). The BAP outlines the actions the FDA plans to take to promote biosimilar and interchangeable product development. Biosimilars are biological products that are highly similar to and have no clinically meaningful differences from existing FDA-approved biologics, known as "reference products." Interchangeable products must meet additional requirements to show that they are expected to produce the same clinical result as the reference product in any given patient. Like generic versions of other drugs, biosimilars are intended to increase access to therapies and promote biologic competition.

With the BAP, the FDA seeks to create a more competitive biosimilar market and provide greater incentives for companies to pursue biosimilar development, because the agency's research suggests that increasing the number of marketed biosimilars will yield significant savings compared to the current cost of biologics. Gottlieb described the situation, "While less than two percent of Americans use biologics, they represent 40 percent of total spending on prescription drugs. So, enabling a path to competition for biologics from biosimilars is a key to reducing costs and to facilitating more innovation."

The BAP highlights four key areas for action:

    1. Improving the efficiency of the biosimilar and interchangeable product development and approval process;
    2. Maximizing scientific and regulatory clarity for the biosimilar product development community;
    3. Developing effective communications to improve understanding of biosimilars among patients, clinicians, and payors; and
    4. Supporting market competition by reducing gaming of FDA requirements or other attempts to unfairly delay competition.

To achieve the goals outlined in the BAP, the FDA will release guidances on biosimilar development, develop new review templates specific to biosimilar products, provide educational materials on biosimilars, and hold a public hearing in September to gather input from stakeholders. The agency has also created the Office of Therapeutic Biologics and Biosimilars within the Office of New Drugs to better support biosimilar development. Though the BAP covers a wide range of actions, the agency acknowledges that some barriers to marketing biosimilars are outside of their purview. Coordination with other agencies like the Federal Trade Commission will be needed to fully address the issues around biosimilar development.

Biologics have revolutionized cancer treatment. The first biosimilar products for cancer biologics were approved in 2017 (bevacizumab-awwb and trastuzumab-dkst). As patent protection expires for more cancer treatment biologics, biosimilar developers will have an opportunity to enter the market. Through the BAP, the FDA is trying to foster a more robust biosimilar market that has the potential to reduce the cost burden cancer patients face.

To learn more about biosimilars, listen to our publicly available webcast of the AACR Annual Meeting 2018 Regulatory Science and Policy Educational Session: Biosimilars: Biological Science, Regulatory Science, and Clinical Practice featuring insights from academia, industry, and the FDA.

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White House Announces Nomination for Director of the Office of Science and Technology Policy    

The Trump administration has announced its intention to nominate Kelvin Droegemeier, PhD, as director of the Office of Science and Technology Policy (OSTP). Droegemeier is an accomplished meteorologist who has been at University of Oklahoma for more than 30 years and currently serves as vice president for research, Regents' professor of meteorology, Weathernews chair emeritus, and Roger and Sherry Teigen presidential professor. Droegemeier’s academic research has focused on understanding severe weather dynamics and improving weather prediction, particularly for thunderstorms and tornados. His contributions to the field include helping develop the use of supercomputers in atmospheric modeling.

In addition to his strong scientific credentials, Droegemeier has also acted in a high-level advisory capacity to state and federal government entities. He served on the National Science Board of the National Science Foundation and was elected as vice chair of the board in 2012. Droegemeier has been part of an Oklahoma state science advisory panel and has advised Representative Jim Bridenstine (R-OK). He also played a role in crafting bipartisan federal legislation to improve weather forecasting. Colleagues have described Droegemeier as an exceptional scientist who is politically-savvy and understands how to navigate the federal bureaucracy.

If confirmed, significant challenges will face Droegemeier as head of OSTP and as a science advisor to President Trump. His nomination comes later into the president’s term than previous OSTP directors, giving him comparatively less time to establish staff and strategic priorities for the office. In addition, Droegemeier will lead OSTP within an administration that has requested substantial annual budget cuts to several scientific agencies and where senior officials have expressed skepticism of science. Many in the scientific community have high hopes that Droegemeier can help provide a strong scientific voice to funding and policy discussions moving forward in the Trump administration.

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AACR Responds to Three Tobacco-Focused FDA Advanced Notices of Proposed Rulemaking     

In July 2017, the newly confirmed U.S. Food and Drug Administration (FDA) Commissioner, Scott Gottlieb, MD, announced a comprehensive regulatory plan to shift the trajectory of tobacco-related disease and mortality. This plan focused on placing nicotine addiction at the center of the agency's tobacco regulatory efforts. In his announcement, Dr. Gottlieb stated that "The overwhelming amount of death and disease attributable to tobacco is caused by addiction to cigarettes –  the only legal consumer product that, when used as intended, will kill half of all long-term users." He continued to state that part of this nicotine-focused plan was that the agency would release three Advanced Notices of Proposed Rulemaking (ANPRMs). ANPRMs were subsequently released regarding: the potential public health benefits and possible adverse effects of lowing nicotine in combustible cigarettes; restrictions on flavors in tobacco products; and regulation of premium cigars. In July 2018, the AACR's Tobacco and Cancer Subcommittee, chaired by Roy S. Herbst, MD, PhD, partnered with the American Society of Clinical Oncology's (ASCO) Tobacco Cessation and Control Subcommittee to respond to these ANPRMs.

In the first ANPRM, Tobacco Product Standard for Nicotine Level of Combusted Cigarettes, the AACR and ASCO recommended based on available scientific evidence that the maximum nicotine level of combusted cigarettes be no greater than 0.4 mg nicotine/g tobacco. This dose of nicotine has been demonstrated to be less reinforcing of addiction than typical nicotine content cigarettes in current smokers, in vulnerable populations (e.g., those with psychiatric conditions), and in youth. Additionally, it was recommended that this standard be applied to all combusted nicotine products (cigars, pipe tobacco, roll-your-own tobacco, etc.) in additional to traditional cigarettes. The AACR and ASCO advised the FDA to carry out nicotine reduction in a single step as opposed to a stepped-down approach with progressively lower nicotine levels. The single step approach has been shown to lead to a more rapid reduction in exposure to toxicants, reduced dependence, and increased cigarette-free days. The comments provided experimental data showing that significant compensatory smoking did not occur with 0.4 mg nicotine/g tobacco cigarettes. Finally, the AACR and ASCO strongly recommended that less harmful nicotine replacement therapies should be available and accessible to smokers to minimize nicotine withdrawal symptoms.

In the second ANPRM, Regulation of Flavors in Tobacco Products, the FDA asked for data and comments on how flavors attract youth to initiate tobacco product use and whether certain flavors may help adult cigarette smokers reduce cigarette use and switch to potentially less harmful products. The AACR and ASCO responded by stating: "The AACR and ASCO recommend that flavorings designed for these products undergo FDA review to determine their safety for inhalation, appropriateness for current smokers seeking to quit using tobacco products and that the product is not designed to attract nor is marketed to children." The AACR reiterated this message on Capitol Hill during our recent briefing, E-cigarettes: Striking a Balance Between Preventing Youth Nicotine Addiction and Helping Current Smokers Quit Combustible Cigarettes

In the final ANPRM, Regulation of Premium Cigars, the AACR and ASCO commented that scientific evidence shows that premium cigars cause substantial harm to those who use them and carry many of the same health risks as cigarette smoking. It was recommended that premium cigars should, therefore, be similarly regulated by the agency. The AACR and ASCO made the specific recommendation that in addition to other factors defining cigars as "premium" (e.g., manufacturing processes, cost, etc.), the FDA consider setting a limit on the pH of premium cigars, e.g., premium cigars tend to have a high pH (~ 6.0). A justification for this pH standard recommendation is that the tobacco industry may try to label something with a lower pH (i.e., lower than 6.0) as a "premium cigar" but it may have a greater abuse liability relative to the other premium cigar (higher pH) products. If a pH threshold (e.g., > 6.5) is considered by the FDA, the toxicity, abuse liability, and pattern of use of associated with this standard would need to be determined.

You can read the three comment letters here.

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Capitol Corner: AACR Interviews Members of Congress   

Every month, the AACR will be interviewing members of Congress to get their personal story and views on cancer research. This month, we interviewed Senator John Boozman (R-Arkansas) and Congressman Steve Stivers (R-Ohio)

Read our previous interviews with Senator Maggie Hassan (D-New Hampshire), Congresswoman Kristi Noem (R-South Dakota), Senator Johnny Isakson (R-Georgia), Congressman Joseph Crowley (D-New York), Senator Jon Tester (D-Montana), Congressman Dave Reichert (R-Washington), Senator Gary Peters (D-Michigan), Congressman Charlie Crist (D-Florida), Senator Susan Collins (R-Maine), Congressman Dwight Evans (D-Pennsylvania), Senator Chuck Grassley(R-Iowa), Senator Tammy Baldwin (D-Wisconsin), Congressman Brett Guthrie (R-Kentucky) and Congresswoman Suzan DelBene (D-Washington).

Senator John Boozman (R-Arkansas)    

Can you share with our readers, many of whom are cancer survivors, advocates, and researchers, your personal connection to cancer?

Like so many others, cancer has touched my life. My father and father-in-law both died from prostate cancer. My mother battled breast cancer.

How has the experience of dealing with cancer in your community, both personally and from stories you've heard from your constituents, shaped your views as a policymaker and a public official?

As a trained medical care provider, I've seen how innovation helps save lives. I believe that we need to invest in research that can promote new advancements to prevent and fight cancer.

As member of the powerful Senate Committee on Appropriations and Senate Budget Committee, you supported FY 2017 appropriations that provided the National Institutes of Health (NIH) with a $2 billion increase in funding. You visited the NIH in June of last year as a part of the largest Senate delegation ever to visit the NIH at one time. The committee last year approved another $2 billion increase to the NIH budget for FY 2018. What would you say to your colleagues in the legislative branch about how important federal investment in medical research is to our nation?

I'm proud to support increases made to NIH funding because the work done here leads to medical advances, prevention, new ways to treat patients and cures for life threatening diseases. This is a responsible investment that will help contain growing health care costs and lead to longer, healthier lives for all Americans. This is good for patients as well as advancing our nation's innovation into lifesaving treatments.

How can groups like the AACR and patient advocates best communicate the importance of medical research to the members of Congress? Do you think we have made progress in terms of raising awareness of the importance of National Institutes of Health (NIH) funding to saving lives and helping the American economy?

Over the past several years, advocates for NIH like AACR have done a great job in communicating its importance to members of Congress and it's crucial those efforts continue. Sharing stories of how research is improving the quality of life for patients and giving hope to families are great ways to show the importance of medical research. With the increase in NIH funding, it's clear that AACR's outreach is making an impact.

Can you tell us more about other efforts—legislation and otherwise—that you have worked on in support of better prevention, detection and treatment of cancer?

I've long supported efforts to advance research to find a cure for cancer. I was proud to support Senate passage of the 21st Century Cures Act. This bill was signed into law in 2016 and authorizes $1.8 billion over seven years for the National Cancer Moonshot Initiative to accelerate research efforts, make new therapies available to patients and add new prevention and detection tools to our war chest to fight and find a cure for cancer.

I'm pleased that crucial research is being done at the University of Arkansas for Medical Sciences (UAMS). I was honored to see UAMS' cancer research labs and look forward to strengthening cancer research in Arkansas.

I routinely meet with Arkansans who advocate on behalf of cancer research funding and I'm pleased to support their requests to encourage my fellow Senators to support investments in research.

The AACR is the world's first and largest organization dedicated to every aspect of high quality cancer research. The AACR has 40,000 members across all states, as well as members in over 120 countries. Do you have anything you would like to say to the AACR and our scientists and physicians who have dedicated their careers to making progress against cancer?

The work AACR does is truly important. There is no substitute for your dedication in the fight against cancer. I applaud your advocacy and research to prevent and cure cancer. The good news is this is a shared goal. You are giving hope to patients, their families and researchers who are committed to making progress and ultimately finding a cure.

Congressman Steve Stivers (R-Ohio)    

Can you share with our readers, many of whom are cancer patients, advocates, and researchers, your personal connection to cancer?

Unfortunately, most everyone today has someone in their life that has been affected by cancer. My dad had cancer and I have had multiple friends that have had cancer. I even have a member of my staff that is a cancer survivor.

How has the experience of dealing with cancer in your community, both personally and from stories you've heard from your constituents, shaped your views as a policymaker and a public official?

From both my personal connections to cancer and the stories I hear from constituents who I meet with, it is clear that we need to support additional funding and research to find more effective treatments – and ultimately a cure – for cancer patients.

You're a co-chair of the Biomedical Research Caucus and a member of the Congressional Caucus on the Deadliest Cancers. You've supported NIH funding increases as well as the 21st Century Cures Act. Why do you believe that NIH funding is so important? What would you say to your colleagues in the legislative branch who may not understand as you do how important federal investment in medical research is to our nation?

Funding the NIH is important because their work is some of our best hope for finding cures, improving treatments, and gaining a better understanding of diseases that affect many Americans. Investing in medical research can literally save lives, and fortunately many of my colleagues also understand the importance of these efforts.

How can groups like the AACR and patient advocates best communicate the importance of medical research to the members of Congress? Do you think we have made progress in terms of raising awareness of the importance of National Institutes of Health (NIH) funding to saving lives and helping the American economy?

The best way to communicate the importance of medical research to members of Congress is to meet and develop relationships with the members and their staff. The AACR and patient advocates should make it a priority to visit member offices to share their stories on why medical research is so important. While we have made progress, there is still work to be done to highlight the importance of the NIH.

Can you tell us more about other efforts—legislation and otherwise—that you have worked on or are currently working on in support of better prevention, detection and treatment of cancer?

In the most recent budget agreement, Congress voted to provide additional funds for the NIH – which is the most important step we can take to support better prevention, detection, and treatment efforts. I am also a cosponsor of the Cancer Drug Parity Act, legislation which will help patients by establishing cost parity for cancer medications, whether administered by a health care provider or self-administered. Moreover, I am also a member of the Congressional Childhood Cancer Caucus, which works to raise awareness about pediatric cancer and advocate in support of policies to help children.

The AACR is the world's first and largest organization dedicated to every aspect of high quality cancer research. The AACR has 40,000 members across all states as well as members in over 120 countries. Do you have anything you would like to say to the AACR and our scientists and physicians who have dedicated their careers to making progress against cancer?

I want to thank the AACR and everyone who is dedicated to the important cause of finding a cure to cancer. I stand with you, and will continue working to ensure medical researchers have the support they need to find cures and save lives.

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FDA-AACR Workshop: Non-clinical Models for Safety Assessment of Immuno-oncology Products   

On Sept. 6, 2018, the AACR and FDA will host a workshop in Washington, D.C., to discuss current challenges and opportunities for non-clinical models to predict toxicities of immune checkpoint inhibitors and stimulators in patients. Registration is now open for participation in-person or via webcast.

The development of non-clinical models that can predict toxicities of immune checkpoint inhibitors and stimulators in patients is an extremely important and timely issue for the cancer community. There are 50 agents targeting PD-1 or PD-L1 in clinical development, and more than 1100 trials combining anti-PD-1/L1 agents with other therapies, yet retrospective analyses indicate that animal toxicity for many of these agents is minimal and does not predict adverse effects in patients.

In addition, the recent observation of worse overall survival in two randomized trials that evaluated anti-PD-1/L1 agents in combination with immunomodulatory drugs underscores the need for cross-sector collaboration in this area. With advances in nonclinical models to study the pharmacodynamics of immune checkpoint inhibitors and stimulators, the question remains whether any of these models could be adapted to assess the safety of immuno-oncology products.

Access the draft agenda, and register for in-person or webcast participation.

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National Academies of Science, Engineering, and Medicine Release Consensus Study Report on Open Science    

Open science is frequently defined as an endeavor to make scientific data, outputs, tools and publications available to all stakeholders in society including the public. Open science can play an important role in accelerating progress in biomedical research. The benefits of data and knowledge-sharing include improved rigor and reproducibility, enhanced ability to identify and take advantage of multidisciplinary opportunities, and faster knowledge dissemination. Open science can also facilitate large scale projects, maximize resource use, and increase public engagement. In recent years, substantial progress has been made to promote open access, though there is still room for improvement and significant challenges remain. The National Academies of Science, Engineering, and Medicine recently released a consensus report establishing a framework for "open science by design" in recognition of the need for continued progress on open science standards, tools, and practices.

While 21st century technology has brought significant opportunities in terms of data, code and knowledge sharing, there are still many obstacles preventing further openness in science. Open access to peer-reviewed publications can be limited as many are often only available via subscription, thereby restricting access for the public and researchers at institutions with limited resources. Challenges also arise around data sharing in many domains because of infrastructure gaps and the high cost of developing and maintaining data sharing platforms. Further, incentives for researchers to share their data and code are often either lacking or actively discouraged for a variety of reasons. Some of the barriers to sharing are unavoidable due to privacy and intellectual property concerns, but many impediments to openness can be overcome through deliberate design of systems and standards to promote openness.

The 2018 National Academies report outlines a comprehensive framework for open science by design. The framework lays out a vision for a system where scientists both contribute to and utilize open resources at all stages of the research process. During provocation researchers could use open resources to explore ideas and collaborative opportunities. Then, as researchers lay out study plans, they would also plan for how to both use and share results and tools in a manner consistent with FAIR (Findable-Accessible-Interoperable-Reusable) standards. Scientists would then use and contribute to open resources during the knowledge-generation, validation, and dissemination stages of their research projects. Finally, scientists would ensure long-term access of research outputs and tools in FAIR archives.

Taking advantage of new technologies and computing capabilities to improve open science will take deliberate and sustained action. The National Academies report is a tremendous resource for the scientific community that puts forward clear and impactful recommendations for action by funders, research institutions, scientific societies, and individual scientists. Such concerted action by the community will help drive improvements in the way science is conducted in the 21st century to accelerate innovation and our ability to ensure new, evidence-based health interventions reach the people who need them.

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FDA Aims to Increase Efficiency with Real-Time Oncology Review and Assessment Aid      

In July, the U.S. Food and Drug Administration (FDA) approved supplemental indications for the drug ribociclib to treat hormone receptor-positive, human epidermal growth factor receptor 2-negative advanced or metastatic breast cancer. These indication approvals are the first granted by the Agency as part of new pilot programs within the Oncology Center of Excellence: Real-Time Oncology Review (RTOR) and Assessment Aid. As a direct result of these pilot programs, these indications for ribociclib were approved on the same day the application was officially filed with the Agency. 

The RTOR program allows the FDA to start analyzing sponsor-provided data as soon as data collection is complete and before the full application is filed. In this context, a sponsor is a person or organization who takes responsibility for and initiates a clinical investigation. The agency and sponsor may now discuss the data leading up to the submission of the application. This discussion provides the sponsor an opportunity to respond to areas of concern identified by the agency before the application is submitted. This more interactive process is expected to increase review efficiency.

Oncology Center of Excellence Director Richard Pazdur, MD, said of the ROTR, "This evaluation is meant to be an it­erative process aimed at maximizing the interactions of the FDA staff and sponsor. In essence, it was what Break­through Designation was all about— early, frequent, and substantive inter­actions aimed at the problems that are of concern to the agency. The real-time review is not only about expediting important applications, but ensuring quality evaluations are performed uti­lizing the resources of both the FDA and sponsor."

The Assessment Aid program is a template used by sponsors to organize their submissions to better facilitate the FDA's review of the application. This template allows the agency to add its assessment to the same file submitted by the sponsor. The goal is for the templated approach to reduce administrative paperwork and give reviewers the ability to better focus on the key details of the application. Of the Assessment Aid, Pazdur noted, "This concise, single document clearly shows areas of agreement between the FDA and the sponsor, and areas where the FDA has concerns. It avoids unneces­sary repetition and duplication of data and analyses found both in the spon­sor's submission and the FDA's written review. Less is more in this situation."

Currently, these pilots are focused on supplemental applications, but the goal is to expand them to all new oncology drug applications. As indicated by the speed of ribociclib's approval, this new review paradigm has the potential to increase the efficiency of oncology drug review and get new safe and effective therapies to patients faster.

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Australia Wins Major Trade Dispute over Tobacco Plain Packaging Law  

In 2010, Australia introduced a law that would ban logos on cigarette packaging and require that these products be sold in bland, olive-colored packs with brand names printed in small, standardized fonts. Australian lawmakers concluded that outlawing common cigarette manufacturer marketing practices could make purchasing tobacco products less appealing thereby improving public health.

Following the 2010 law's passage and prompted by the tobacco industry, the nations of Cuba, Indonesia, Honduras, and the Dominican Republic filed a complaint with the World Trade Organization (WTO). In their complaint, these nations stated that Australia's law had unjustifiably infringed upon tobacco trademarks and had violated intellectual property rights. These nations also stated that there were alternative measures to the plain packaging law that would be equally effective in improving public health.

On June 28, 2018, the WTO issued a landmark ruling rejecting this complaint. WTO judges dismissed the argument that the Australia law violated trademark and intellectual property rights. Additionally, the judges ruled that the law would improve public health by reducing the use of tobacco products. Following the ruling, Honduras has indicated that it is likely to appeal and Indonesia is weighing its legal option.

This World Health Organization praised the ruling, stating that this is a positive step in preventing the tobacco industry from blocking tobacco control efforts and that this victory is likely to accelerate the implementation of similar packaging laws around the world. Currently, six other countries have plain packaging laws: Hungary, Ireland, France, New Zealand, Norway, and Great Britain. Another six countries, Burkina Faso, Canada, Georgia, Romania, Slovenia, and Thailand, have passed laws that are yet to be implemented.

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